Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)

April 21, 2026 updated by: Avidity Biosciences, Inc.

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

AOC 1020-CS1 is a first-in-human, 3-part, multi-center, Phase 1/2, randomized, double-blind, placebo-controlled study designed to evaluate safety, tolerability, pharmacokinetics and to explore pharmacodynamics and efficacy of single and multiple-doses of AOC 1020 administered intravenously in participants with FSHD Type 1 (FSHD1) and FSHD Type 2 (FSHD2).

Cohort A comprises a placebo-controlled dose titration cohort (Cohort A1) which includes a nested single and multiple dose schedule. Cohort B comprises a placebo-controlled, nested single ascending dose (SAD)/multiple ascending dose (MAD) cohort (Cohort B1). Cohort C comprises a randomized, placebo-controlled, expansion cohort (Cohort C1). For each of Cohorts A, B, and C the study duration is 12 months as the active treatment period is approximately 9 months for Cohorts A & B and approximately 10.5 months for Cohort C followed by a 12-week follow-up period for Cohorts A & B and a 7-week follow-up period for Cohort C. Once participants have completed active treatment with follow-up through 12 months, they may have the option to participate in a planned open-label extension. If patients do not immediately roll over into the open-label extension study or decline participation, they will be followed for 18 weeks after their last dose of study medication.

Study Type

Interventional

Enrollment (Actual)

90

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1Y 4E9
        • University of Ottawa
  • United Kingdom
      • London, United Kingdom, WIT 7HA
        • University College London
      • Sheffield, United Kingdom, S10 2TN
        • University of Sheffield
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • University of California Los Angeles
      • Palo Alto, California, United States, 94304
        • Stanford University
      • San Diego, California, United States, 92093
        • University of California San Diego
    • Colorado
      • Denver, Colorado, United States, 80045
        • University of Colorado
    • Florida
      • Gainesville, Florida, United States, 32608
        • University of Florida
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Rare Disease Research
    • Kansas
      • Kansas City, Kansas, United States, 66205
        • Kansas University Medical Center
    • New York
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center
    • North Carolina
      • Durham, North Carolina, United States, 27708
        • Duke University
    • Ohio
      • Columbus, Ohio, United States, 43221
        • Ohio State University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
    • Texas
      • Dallas, Texas, United States, 75390
        • University of Texas Southwestern
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University
    • Washington
      • Seattle, Washington, United States, 98104
        • University of Washington

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor)
  • Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
  • At least 1 muscle region suitable for biopsy (testing provided by Sponsor)
  • Muscle weakness in both upper and lower body, as determined by Investigator

Exclusion Criteria:

  • Pregnant or intends to become pregnant while on study, or active breastfeeding
  • Unwilling or unable to continue to comply with contraceptive requirements
  • Body mass index (BMI) >35.0 kg/m2 at Screening
  • History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study
  • History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy
  • Anticipated survival less than 2 years
  • Blood or plasma donation within 16 weeks of Study Day 1
  • Any contraindication to MRI
  • Any abnormal lab values, conditions or diseases that, in the opinion of the investigator or Sponsor, would make the participant unsuitable for the study or could interfere with participation or completion of the study
  • Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AOC 1020 Regimen 1
Cohort A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months
Drug: AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion
Experimental: AOC 1020 Regimen 2
Cohort B1: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months
Drug: AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion
Experimental: AOC 1020 Regimen 3
Cohort C: AOC 1020 Dose Regimen 3; Eight doses administered intravenously over approximately 10 months
Drug: AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion
Placebo Comparator: Placebo (Saline) Regimen 1
Cohort A & B: Placebo; Five doses administered intravenously over 9 months
Drug: Placebo
Placebo will be administered via intravenous (IV) infusion
Other Names:
  • Saline
Placebo Comparator: Placebo (Saline) Regimen 2
Cohort C: Placebo; Eight doses administered intravenously over approximately 10 months
Drug: Placebo
Placebo will be administered via intravenous (IV) infusion
Other Names:
  • Saline

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of treatment-emergent adverse events (Cohorts A & B)
Time Frame: Through study completion, up to Day 365
Through study completion, up to Day 365
Change in plasma KHDC1L (Part C)
Time Frame: Across months 3 to 12
Ratio to Baseline
Across months 3 to 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Time Frame: Through study completion; up to Day 365
Observed maximum concentration
Through study completion; up to Day 365
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Time Frame: Through study completion; up to Day 365
Observed half-life
Through study completion; up to Day 365
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Time Frame: Through study completion; up to Day 365
Observed area under the curve
Through study completion; up to Day 365
Muscle drug concentration (Cohorts A & B)
Time Frame: Day 120
Concentration of siRNA component in skeletal muscle
Day 120
Change in circulating creatine kinase (Cohort C)
Time Frame: Across months 3 to 12
Ratio to Baseline
Across months 3 to 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Avidity Biosciences, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 4, 2023

Primary Completion (Actual)

March 18, 2026

Study Completion (Actual)

March 20, 2026

Study Registration Dates

First Submitted

February 17, 2023

First Submitted That Met QC Criteria

February 27, 2023

First Posted (Actual)

February 28, 2023

Study Record Updates

Last Update Posted (Actual)

April 24, 2026

Last Update Submitted That Met QC Criteria

April 21, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AOC 1020-CS1
  • 2022-002704-20 (EudraCT Number)
  • 2022-502096-32-00 (Other Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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